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Global Blood Therapeutics is part of our Disease Eradication portfolio.

The CDC estimates that there are 100,000 people who are currently affected by sickle cell disease

The estimated cost of medical care for the roughly 70,000 individuals with sickle cell disease in the US exceeds $1.1 billion

When an illness is serious, the worst thing a patient can hear is that there are few options for care. This isn’t necessarily a doctor’s fault, of course, although that isn’t much comfort to the patient. All she or he can do is hope that the treatments available for few symptoms can provide solace in some way, and that the recommendations for managing a new lifestyle actually work. And perhaps a trial drug can become available to target the exact cause of discomfort in time.

Obviously, those solutions aren’t exactly good enough when someone is experiencing pain. But if a patient’s particular pain is related to sickle cell disease, that is what he or she has to deal with. According to the Centers for Disease Control and Prevention, there are millions of people throughout the world who are affected by this disease, which describes a group of inherited blood cell disorders. The exact number of cases in the United States is unknown, but the CDC estimates that there are 100,000 people who are currently affected by the disease. It’s the most common genetic disorder in the country, and 1,000 babies are born with it every year. It should also be noted that this disease especially impacts African American babies, and 1 in 365 are born with the symptoms. Overall, the disease is most prevalent among African American, Middle Eastern, Mediterranean, and Hispanic populations.

Sickle cell disease is chronic and incurable, although it can be treated with careful precaution. People who were diagnosed at birth have to receive ongoing care from teams of doctors that treat them from childhood into adulthood, and most live into their 40s and 50s. And while that age range is an improvement from the past, companies like Global Blood Therapeutics are working to extend it even further. Since it was founded in San Francisco seven years ago, this company has been making strides in the discovery, development, and implementation of a sole drug for sickle cell disease. This pill, voxelotor, is in the late stages of development, and the FDA gave it a “breakthrough therapy” designation early this year. Because of that and other promising factors, the company has a market capitalization of about $2.8 billion.

Before we dive into the work that Global Blood Therapeutics is up to, let’s chat a little more about the details of this disease.

It’s in your blood

So what exactly do we mean when we’re talking about sickle cell disease? Well, for starters, we’re talking about something that is with someone for life, and cannot be developed as an adult. Sickle cell disease is passed on from the genes of parents to their children, and is classified by abnormal hemoglobin in red blood cells. Hemoglobin is a protein that helps those cells carry oxygen from the lungs through the tissues in the body, and the roundness of a “normal” red blood cell allows them to pass without incident. However, “sickle” hemoglobin alters the round blood cells into crescent shapes, which can attach to vessel walls and cause a blockage. This stops oxygen from reaching certain parts of the body, infects immediate pain, and requires a hospital visit. In can also cause strokes.

As the child grows with sickle cell disease, they may not experience pain on a daily basis. But that pain can be chronic as an adult, and can affect the whole system of the body: the brain, lungs, heart, eyes, and more, and especially the spleen. Furthermore, adults with sickle cell disease don’t have the same robust healthcare options as children, and the struggle to switch from pediatric supervision to adult care can exacerbate symptoms. Adults with the disease have to worry about common concerns — a balanced diet and regular exercise — but they also need to be aware of abrupt shifts in temperature and dehydration. The pain can be so severe that it may also lead to depression.

Global Blood Therapeutics is focused on one medication that can help hemoglobin retain oxygen, otherwise allowing red blood cells to keep their circular shape. This is the basic premise of voxelotor, the once-daily oral pill that this biotech company is exclusively focused on developing for people of all ages. Its ongoing studies are classified under the HOPE study, which is currently in its third phase. The purpose of the study evaluates the success rates of voxelotor in decreasing the number of sickle cells within the bloodstream over time, with the ultimate goals of decreasing the painful symptoms of the disease on a daily basis and extending the lifespans of those affected. HOPE also has a child-based study for those between six and 17.

So far, Global Blood Therapeutics is seeing success and growing acceptance around the world. The FDA has given vexelotor three important labels — the Fast Track, Orphan Drug, and Rare Pediatric Disease designations — to spur further testing and hopefully greater access, and the European Medicines Agency has put the drug in its Priority Medicines program. Lastly, the European Commission has determined that voxelotor can be called an “orphan medicinal product,” which provides incentive to continue its progress.

In its year-end financial results, Global Blood Therapeutics noted these judgments as “momentum” that will help the company push forward on its mission to confidently release this drug in the future. This year, with help from net proceeds totaling about $111 million, it will collect even more data and grow its pipeline to include more research and business developments.

And while those goals are noble, those aren’t the only things inspiring the progress of Global Blood Therapeutics.

More money, more problems

While managing pain and increasing life expectancy are the company’s premier drivers, it is also aware that the currently available treatments are pricey. In fact, Global Blood Therapeutics lists the total annual cost of an adult patient with the most common form of sickle cell disease at more than $200,000, according to a study. This comes out to more than $8 million in associated hospital expenses over the course of an ideal 50-year lifespan.

In 2009, a study at the University of Florida combed through the expenses of 4,294 relevant patients in the state’s Medicaid program and found that the average patient saw a monthly cost of $1,946. The numbers varied across age groups.

Reuters picked up this study when it was published in the American Journal of Hematology and said, “By their calculations, the estimated cost of medical care for the roughly 70,000 individuals with sickle cell disease in the US ‘exceeds $1.1 billion.’”

It is Global Blood Therapeutics’ hope that this extremely large financial burden can be lessened by its revolutionary oral drug. Time will tell if that is the case, but if it is, those with sickle cell disease can see future improvements in their health and their bottom lines. That combination can lead to a life with more available options than those with sickle cell disease have had the opportunity to imagine. And when you’re a patient who is sick, options are exactly what you want to hear.

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